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WO2016168592A3 - Compositions for modulating c9orf72 expression - Google Patents

Compositions for modulating c9orf72 expression Download PDF

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Publication number
WO2016168592A3
WO2016168592A3 PCT/US2016/027747 US2016027747W WO2016168592A3 WO 2016168592 A3 WO2016168592 A3 WO 2016168592A3 US 2016027747 W US2016027747 W US 2016027747W WO 2016168592 A3 WO2016168592 A3 WO 2016168592A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
c9orf72 expression
modulating
modulating c9orf72
methods
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2016/027747
Other languages
French (fr)
Other versions
WO2016168592A2 (en
Inventor
Frank Rigo
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority to RU2017139121A priority Critical patent/RU2736574C2/en
Priority to BR112017016068-4A priority patent/BR112017016068A2/en
Priority to CN201680016814.1A priority patent/CN107427532B/en
Priority to HK18108871.7A priority patent/HK1250255B/en
Priority to LTEP16780833.6T priority patent/LT3283080T/en
Priority to RS20200535A priority patent/RS60230B1/en
Priority to AU2016249002A priority patent/AU2016249002B2/en
Priority to NZ733882A priority patent/NZ733882A/en
Priority to DK16780833.6T priority patent/DK3283080T3/en
Priority to CA2978103A priority patent/CA2978103A1/en
Priority to MX2017013199A priority patent/MX366128B/en
Priority to KR1020207010994A priority patent/KR102258516B1/en
Priority to MYPI2017703869A priority patent/MY181815A/en
Priority to JP2017546618A priority patent/JP6606557B2/en
Priority to KR1020177032588A priority patent/KR102104163B1/en
Priority to MX2019007834A priority patent/MX385674B/en
Priority to SI201630693T priority patent/SI3283080T1/en
Priority to HRP20200843TT priority patent/HRP20200843T1/en
Priority to EP16780833.6A priority patent/EP3283080B1/en
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Priority to ES16780833T priority patent/ES2791995T3/en
Priority to EP20163264.3A priority patent/EP3722424A1/en
Priority to PL16780833T priority patent/PL3283080T3/en
Priority to SG11201708468YA priority patent/SG11201708468YA/en
Priority to SM20200165T priority patent/SMT202000165T1/en
Publication of WO2016168592A2 publication Critical patent/WO2016168592A2/en
Publication of WO2016168592A3 publication Critical patent/WO2016168592A3/en
Priority to IL253557A priority patent/IL253557B/en
Priority to CONC2017/0007671A priority patent/CO2017007671A2/en
Priority to PH12017501875A priority patent/PH12017501875B1/en
Priority to SA520412450A priority patent/SA520412450B1/en
Priority to SA517390168A priority patent/SA517390168B1/en
Anticipated expiration legal-status Critical
Priority to SA520412451A priority patent/SA520412451B1/en
Priority to AU2019250156A priority patent/AU2019250156B2/en
Priority to PH12019502724A priority patent/PH12019502724A1/en
Priority to CY20201100520T priority patent/CY1122981T1/en
Priority to IL277624A priority patent/IL277624A/en
Ceased legal-status Critical Current

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/22—Anxiolytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

Disclosed herein are compositions and methods for reducing expression of C90RF72 mRNA and protein in an animal. Such methods are useful to treat, prevent, ameliorate, or slow progression of neurodegenerative diseases in an individual in need thereof.
PCT/US2016/027747 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression Ceased WO2016168592A2 (en)

Priority Applications (34)

Application Number Priority Date Filing Date Title
EP16780833.6A EP3283080B1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
CN201680016814.1A CN107427532B (en) 2015-04-16 2016-04-15 Compositions for modulating expression of C9ORF72
HK18108871.7A HK1250255B (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
LTEP16780833.6T LT3283080T (en) 2015-04-16 2016-04-15 C9ORF72 EXPRESSION MODULATION COMPOSITIONS
RS20200535A RS60230B1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
AU2016249002A AU2016249002B2 (en) 2015-04-16 2016-04-15 Compositions for modulating C90RF72 expression
NZ733882A NZ733882A (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
DK16780833.6T DK3283080T3 (en) 2015-04-16 2016-04-15 COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION
CA2978103A CA2978103A1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
MX2017013199A MX366128B (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression.
KR1020207010994A KR102258516B1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
MYPI2017703869A MY181815A (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
JP2017546618A JP6606557B2 (en) 2015-04-16 2016-04-15 Composition for modulating C9ORF72 expression
KR1020177032588A KR102104163B1 (en) 2015-04-16 2016-04-15 Composition for modulating C9ORF72 expression
MX2019007834A MX385674B (en) 2015-04-16 2016-04-15 COMPOSITIONS TO MODULATE THE EXPRESSION OF C9ORF72.
BR112017016068-4A BR112017016068A2 (en) 2015-04-16 2016-04-15 c9orf72 expression modulation compositions
HRP20200843TT HRP20200843T1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
RU2017139121A RU2736574C2 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
SI201630693T SI3283080T1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
SG11201708468YA SG11201708468YA (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
EP20163264.3A EP3722424A1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
PL16780833T PL3283080T3 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
ES16780833T ES2791995T3 (en) 2015-04-16 2016-04-15 Compositions to modulate the expression of C90RF72
SM20200165T SMT202000165T1 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
IL253557A IL253557B (en) 2015-04-16 2017-07-18 Compositions for modulating c9orf72 expression
CONC2017/0007671A CO2017007671A2 (en) 2015-04-16 2017-07-28 Compositions to modulate c9orf72 expression
PH12017501875A PH12017501875B1 (en) 2015-04-16 2017-10-13 Compositions for modulating c9orf72 expression
SA520412450A SA520412450B1 (en) 2015-04-16 2017-10-16 Compositions for modulating c9orf72 expression
SA517390168A SA517390168B1 (en) 2015-04-16 2017-10-16 Compositions for modulating c9orf72 expression
SA520412451A SA520412451B1 (en) 2015-04-16 2017-10-16 Compositions for modulating c9orf72 expression
AU2019250156A AU2019250156B2 (en) 2015-04-16 2019-10-16 Compositions for modulating c9orf72 expression
PH12019502724A PH12019502724A1 (en) 2015-04-16 2019-12-02 Compositions for modulating c9orf72 expression
CY20201100520T CY1122981T1 (en) 2015-04-16 2020-06-02 COMPOSITIONS FOR MODULATING THE EXPRESSION OF C9ORF72
IL277624A IL277624A (en) 2015-04-16 2020-09-25 Compositions for modulating c9orf72 expression

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562148691P 2015-04-16 2015-04-16
US62/148,691 2015-04-16
US201562232941P 2015-09-25 2015-09-25
US62/232,941 2015-09-25
US201562239400P 2015-10-09 2015-10-09
US62/239,400 2015-10-09

Publications (2)

Publication Number Publication Date
WO2016168592A2 WO2016168592A2 (en) 2016-10-20
WO2016168592A3 true WO2016168592A3 (en) 2016-11-24

Family

ID=57126261

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2016/027747 Ceased WO2016168592A2 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression

Country Status (30)

Country Link
US (4) US9605263B2 (en)
EP (2) EP3722424A1 (en)
JP (3) JP6606557B2 (en)
KR (2) KR102258516B1 (en)
CN (2) CN107427532B (en)
AU (2) AU2016249002B2 (en)
BR (1) BR112017016068A2 (en)
CA (1) CA2978103A1 (en)
CL (4) CL2017002567A1 (en)
CO (1) CO2017007671A2 (en)
CY (1) CY1122981T1 (en)
DK (1) DK3283080T3 (en)
ES (1) ES2791995T3 (en)
HR (1) HRP20200843T1 (en)
HU (1) HUE049233T2 (en)
IL (2) IL253557B (en)
LT (1) LT3283080T (en)
MX (2) MX385674B (en)
MY (1) MY181815A (en)
NZ (1) NZ733882A (en)
PH (2) PH12017501875B1 (en)
PL (1) PL3283080T3 (en)
PT (1) PT3283080T (en)
RS (1) RS60230B1 (en)
RU (1) RU2736574C2 (en)
SA (3) SA520412451B1 (en)
SG (2) SG11201708468YA (en)
SI (1) SI3283080T1 (en)
SM (1) SMT202000165T1 (en)
WO (1) WO2016168592A2 (en)

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US10428019B2 (en) 2010-09-24 2019-10-01 Wave Life Sciences Ltd. Chiral auxiliaries
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JP6268157B2 (en) 2012-07-13 2018-01-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ Asymmetric auxiliary group
ES2762326T5 (en) 2012-10-15 2023-04-27 Ionis Pharmaceuticals Inc Methods to modulate the expression of C9ORF72
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RU2748426C2 (en) * 2013-10-11 2021-05-25 Ионис Фармасьютикалз, Инк. Composition for modulation of c90rf72 expression
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AU2018316159A1 (en) * 2017-08-08 2020-02-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
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